Help Children with SMA Disease
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Spinal muscular atrophy (SMA) or spinal muscular atrophy is an inherited muscle disease usually occurs when children are very young. In SMA disease, muscle control is impaired. It is the most common genetic cause of infant death: about 600 children in Europe are born each year with SMA, a rare disease that affects muscle function, including breathing, swallowing and movement.
The most severe SMA type 1 affects about half of the affected children: the first signs are visible at birth or in the first few months after birth. Research has made great progress. In particular, the gene therapy drug Zolgensma has shown important results in SMA treatment. It is the most expensive drug in the world: the treatment costs more than 2 million euros.